MT2022-01: MSCs for ALD

Summary

This is a single-institution study to evaluate the use of intravenously administered allogeneic, 3rd party mesenchymal stem cells (IV-MSC) in patients with active, cerebral adrenoleukodystrophy (CALD), as a bridge to hematopoietic stem cell transplant or gene therapy, or in patients who are too advanced for gene therapy or HSCT. The intervention will occur in the time between diagnosis of active CALD and transplant which is currently 8-12 weeks.

Timeline

Start

2023-08-28

Primary completion

2027-05-01

Completion

2029-05-01

Outcome

Outcome not reported

Stopped: “Awaiting supplies”

Drugs

Indications